Verhaart and Aartsma-Rus discuss important advances in the treatment of Duchenne muscular dystrophy. Gene-addition, exon-skipping, stop codon readthrough and genome-editing approaches aim to restore expression of partially functional dystrophin, whereas treatments that target disrupted pathophysiological pathways can improve muscle function.
- Ingrid E. C. Verhaart
- Annemieke Aartsma-Rus